National Institute
on Alcohol Abuse and Alcoholism No. 17 PH 322 July 1992

Treatment Outcome
Research

Treatment outcome research
is designed to answer six basic questions: Is treatment better than no treatment?
Is treatment worse than no treatment? Is one treatment better than another?
If a treatment is effective, is a little just as good as a lot? Does quality
of life change because drinking has changed? Are the benefits of treatment
worth the cost? Although no single study is likely to answer all of these
questions, every medical and behavioral treatment needs to be evaluated along
these lines.

Until the last decade,
alcoholism treatment research lagged behind standard medical and behavioral
treatment research. Randomized controlled trials, the most objective type
of treatment research methodology, were rarely used by alcohol treatment researchers.
Instead, much of the early research suffered from lack of comparison groups,
high rates of attrition, and reliance solely on self-reports of alcohol consumption,
all of which may have biased, or skewed, the results.

Methodology

Controlled trials. Controlled
clinical trials have become a widely accepted method for evaluating treatment
with the least bias (1). This method uses comparison groups, known as controls,
that receive either no treatment or a treatment different from the treatment
under study. Researchers evaluate treatment effectiveness by comparing patient
outcome in the study group to patient outcome in the control group.

Randomization. Differing
patient characteristics can influence the outcome of research--some patients
are more motivated to recover, some are sicker than others, some have more
social support. To distribute these characteristics evenly among comparison
groups, researchers place patients in groups at random, a method known as
randomization. Such randomization produces treatment groups that are likely
to be equivalent in every aspect but the treatment itself.

Blinding. Double-blind
studies, in which both evaluators and patients are unaware of which patients
receive which treatments, keep research results objective (1). When testing
medications, neither patients nor evaluators can distinguish between a placebo
and the actual medication. However, with verbal therapies (common in most
alcoholism treatments), only the evaluators can be blinded.

Followup. A well-done
study follows up all participants, including those who drop out of the study.
Following only patients who remain in a study may exaggerate the effectiveness
of a particular treatment because those who drop out usually do so because
they have relapsed. How long study participants should be followed is a matter
for debate. Because most relapse tends to occur within the first 6 months
after the completion of treatment, some argue that a 6-month followup is sufficient.
Others contend that patients should be followed for as long as 2 years. The
duration of followup usually is determined by the objective of the study and
the financial resources available to support the study.

Outcome measures. To
assess treatment effectiveness, researchers use a variety of outcome measures,
including patient self-reports, to gauge changes in drinking behavior, physical
health status, psychological health status, and social functioning. These
measures must be valid and consistent. Because patient self-reports may be
inaccurate, some researchers recommend that the reports be verified by relatives
or friends close to the patient and/or by periodic laboratory testing of urine,
blood, or breath alcohol levels (2). Because alcohol remains in body fluids
less than 12 hours, a laboratory test that could measure long-term alcohol
consumption is needed. Efforts to develop such a test are in progress. Cu
rrently, the serum gamma-glutamyltransferase (GGT) is being used successfully
by some researchers to corroborate patient self-reports (3).

Where Is Research
Needed?

Many commonly used treatments
have not been adequately evaluated and need to undergo controlled clinical
trials. These trials will not only verify the effectiveness of treatment but
also may help to improve outcome and cost-effectiveness. The highly regarded
approach to alcoholism, Alcoholics Anonymous (AA), has proved difficult to
evaluate. Part of the problem stems from the difficulty of studying AA under
natural conditions and the inability to randomize patient samples due to the
AA tradition of member anonymity (4). A popular treatment approach, the inpatient
"Minnesota Model," uses a philosophy of self-help similar to that of AA (5).
The program combines referral to AA both during inpatient treatment and as
part of an aftercare program. The model relies heavily on counseling (both
personal and family). Few controlled studies have been conducted on the model's
effectiveness (6,7); however, two recent studies, one in the United States
(8) and one in Finland (9), support its effectiveness.

Inpatient Versus
Less Intensive Treatment: What Does Treatment Outcome Research Reveal?

The effectiveness of
inpatient versus less intensive treatment continues to be debated and studies
have been conducted comparing the two at all stages of recovery. In a well-designed
randomized study, Hayashida and colleagues compared detoxification using benzodiazepines
in an inpatient setting and in an outpatient setting and found no difference
in outcome between the two (10). After detoxification, treatment for long-term
recovery can begin. One study compared outcome of an inpatient alcoholism
treatment program with outcome of a day hospital program and found that the
intensive outpatient treatment was as effective as the inpatient treatment
(11). In a controlled clinical trial, Walsh and colleagues compared hospitalization
(including AA) with AA alone for employees at risk of job loss (8). Results
of this study suggest that inpatient rehabilitation produces a more effective
outcome than AA alone. Although these individual studies cannot be compared
directly with one another because they examine quite different treatments,
together they serve as a basis for other controlled studies investigating
intensive versus less intensive treatment.

Treatment outcome research
has provided evidence that other treatments may help reduce drinking among
recovering alcoholics. These include behavioral training such as stress management
therapy, assertiveness and communication skills training, behavioral self-control
training, and behavioral marital therapy. One controlled clinical trial found
that social skills training decreased the duration and severity of relapse
after 1 year in a group of alcoholics (12). Research has been done on acupuncture
therapy; however, two recent studies have produced conflicting results (13,14).

Medications

Two types of medications
have been introduced to reduce drinking: one to deter drinking and another
to reduce the craving for alcohol. A large body of outcome research addresses
the use of disulfiram, a medication that deters drinking. Many studies have
reported favorable results, but most were methodologically flawed. More recent,
better designed studies have not replicated the results of earlier studies.
Fuller and colleagues (15) conducted a well-controlled clinical trial examining
the efficacy of disulfiram. Patients were placed in one of three groups at
random: a treatment group, receiving a daily 250 mg dose of disulfiram; and
two control groups, one receiving a daily 1 mg dose of disulfiram and the
other receiving a vitamin in place of disulfiram. Patients were followed up
seven times over a 1-year period and outcome was measured with self-reports
corroborated by interviews with relatives or friends, and by blood an d urine
tests (15). Although there was no significant difference among the three groups
in terms of total abstinence, the group receiving the 250 mg of disulfiram
reported significantly fewer drinking days than the other two groups. Another
controlled study found disulfiram to be most effective for married patients
when their spouses attempted to ensure that they took their medication (16).

Treatment outcome research
has examined a wide range of other medications, including antidepressants
and lithium. Studies are investigating lithium's effectiveness in treating
alcoholism, independent of its effect on manic depression (i.e., Does lithium
treat alcoholism by treating depression, or does it have a direct effect on
drinking behavior?). However, a well-designed study (17) found lithium ineffective
in treating alcoholics without manic-depressive syndrome.

Other studies are investigating
promising new pharmacotherapies. In preliminary studies, the opiate antagonist,
naltrexone, appears to reduce the frequency of relapse (18,19). If replicated,
such research may lead to pharmacotherapy becoming integral to alcoholism
rehabilitation.

Increasing Efficiency
Through Outcome Research

Research has shown that
alcoholism is a heterogeneous disease that may require multiple methods of
treatment (20). Treatment that works well for one type of alcoholic may not
work for another.

Traditionally, alcoholism
treatment programs have offered patients a mix of treatment approaches. A
promising new strategy involves matching patients to interventions more specific
to their needs. Determining which patients respond best to which treatments
can increase treatment effectiveness. A wide range of patient-treatment matching
effects have already been reported (21).

Efficiency and cost of
alcoholism treatment are important considerations for many patients, third-party
payers, and clinics deciding on a treatment type. As outcome research distinguishes
effective from ineffective treatments, judgments can begin to be made based
on cost (7).

While treatment outcome
research is not a new idea, it is fairly new in the alcohol field. Just as
it would be unthinkable to unleash a new drug therapy for cancer, a new antibiotic
for kidney

disease, or a new contraceptive
without scientific evidence of safety and efficacy, treatments for alcoholism
must be rigorously evaluated to ensure that patients get the best help possible.

Treatment outcome studies
are designed to answer commonsense questions. To determine whether a treatment
accomplishes anything, we have to know how patients who have not received
the treatment fare. Perhaps untreated patients do just as well, implying that
the treatment does not influence outcome at all. Or, perhaps treated patients
do worse.

Research can provide
information that could help reduce the cost and inconvenience of treatment
to patients. If the treatment is helpful, a little bit of it may be as useful
as a lot. We must also determine whether a treatment that appears effective
under ideal circumstances (e.g., good patient compliance and well-trained
staff) will work under "real world" conditions (crowded clinics, varying levels
of staff training, and poor patient compliance).

Treatment outcome research
will support new approaches; alcoholism treatment providers must take the
time to keep up with and apply the results of research in their programs or
practice. Simply put, there is nothing sacred about any of today's treatment
methods. For any disease, we hope that tomorrow's treatment will be better
than today's. The experience and wisdom of the

many caring, competent,
and dedicated alcoholism treatment personnel will continue to be key ingredients
in alcoholism treatment. However, new treatment technologies o r techniques
will improve patient outcome, and it is important that treatment providers
stay abreast of new developments.